OXFORD, UK / ACCESSWIRE / December 3, 2018 / IntraBio Inc., a late-stage biopharmaceutical company developing novel therapies for rare ("orphan") and common neurodegenerative diseases, announced that the US Food and Drug Administration (FDA) has granted a Rare Pediatric Disease Designation (RPDD) to its lead compound series (IB1000s) for the treatment of Niemann-Pick disease Type C (NPC), a rare, debilitating, inherited lysosomal storage disorder that predominately affects pediatric patients.
IntraBio has previously been granted Orphan Medicinal Drug Designation from the European Commission and Orphan Drug Designation from the FDA for IB1000s for the treatment of NPC.
The Rare Pediatric Disease Designation makes IB1000s eligible for, and expedites the request of, a Rare Pediatric Disease Priority Review Voucher (PRV) granted at the time of New Drug Authorization (NDA). This PRV can be redeemed to receive a priority review of a subsequent marketing application for a different product and can be sold or transferred an unlimited number of times. In 2016, four vouchers were sold for an average price of $200 million each.
"The Rare Pediatric Disease Designation for IB1000s emphasizes the need for the development and commercialization of rare-disease drugs," added Mallory Factor, Chairman and Chief Executive Officer of IntraBio Inc. "The designation is an important step in enabling us to develop novel therapeutic options in these areas of high unmet medical needs."
NPC affects 1:100,000 live births and is most commonly caused by dysfunction of the NPC1 protein leading to the accumulation of lipids in lysosomes, resulting in impaired cell function and cell death in various organs, leading to a spectrum of symptoms in NPC patients. The disease typically begins in early childhood and is chronic and progressive in nature; motor and cognitive symptoms become more disabling over the course of the disease, negatively impacting the quality of life. Currently, the average age of death for NPC patients is approximately 10 years, with half of the patients dying before the age of 12.5 years.
IntraBio, with its collaborators, has evaluated the effect of IB1000s in compassionate-use studies in over 175 patients, forming the scientific basis for IB1000s to be further investigated for the treatment of 18 indications, including neurodegenerative diseases and lysosomal storage disorders. Future opportunities to develop the IB1000s series further in additional indications include Lewy Body Dementia (LBD), Parkinsonisms, Restless Leg Syndrome (RLS), Amyotrophic Lateral Sclerosis (ALS), and Multiple Sclerosis (MS), all of which of have high-unmet medical needs.
IntraBio is currently in the process of applying for multi-national clinical trials with its lead asset (IB1001) for the treatment of Niemann-Pick disease Type C (NPC), GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease) and certain inherited Cerebellar Ataxias (CA).
IntraBio Inc. is a biopharmaceutical company with a late-stage drug pipeline including novel treatments for common and rare neurodegenerative diseases. IntraBio's platform results from decades of research and investment at premier universities and institutions worldwide. Its clinical programs leverage the expertise in lysosomal function and intracellular calcium signaling of its scientific founders from the University of Oxford and the University of Munich.
IntraBio's management team and consultants have vast commercial experience and a successful track record of drug development in the USA and Europe. Together, IntraBio's team translates innovative scientific research in the fields of lysosomal biology, autophagy, and neurology into novel drugs for a broad spectrum of genetic and neurodegenerative diseases so to significantly improve the lives of patients and their families.
IntraBio Inc. is a US corporation with its principal laboratories and offices in Oxford, United Kingdom.
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